Tuesday, April 23, 2013

Stem cells and regenerative medicine


Stem cells and Regenerative medicine

 

Every human being would like to have an everlasting healthy life untouched by disease, and ageing process. The starfish’s ability to regenerate its lost arms or the ability of the lizard to regenerate its tail has fascinated both young minds and the wise alike. ‘What if we have an option of replacing a malfunctioning organ with a brand new one’? With Successful heart, kidney, liver, corneal transplantation, mankind today has made a beginning in this direction.  But, these spares from other fellow beings come with limitations. The availability of suitable donors, both the one who is willing to donate and importantly if not more is the necessity of ‘cross match’ between the host and the donor. Thus, shortage of organs and organ rejections are serious limitations. The possibility of making our own organs soon received attention. Science of regenerative medicine is an emerging interdisciplinary area that involves designing customized organs and tissues using one’s own cells (the recipient in this case is the donor himself/herself). It also encompasses the repair of tissues or organs that are damaged in-situ and more recently also is expanded to include the scope of improved function of an organ/ tissue. The key to progress in this direction comes from a combination of the use of cells, materials, engineering and creation of the milieu. Today, the term ‘stem cells’  which was initially proposed for scientific use by Alexander Maksimov, a Russian histologist at the congress of hematological society in Berlin, is a very popular one and attracts significant interests both among the scientific community as well as the general public. There are companies that offer their technology to preserve stem cells for a future application. This might appear at least to some as a more refined way (modern) of the ancient Egyptian quest for eternity. Perhaps this is a field that did not have to take much effort to reach to public, in most cases there was considerable attention even from media which may be quoted as a good example of how research should translate to application, but unfortunately it has led to some ‘unrealistic expectations’ as well.

Stem cells may be understood as a mother cell that have the potential or ability to multiply and differentiate into a more specialized cell  or simply can self renew to produce more stem cells. The well orchestrated and precise transformation of a single cell (zygote) to a multicellular organism has been the initial fuel for scientists working on the possibility of regenerative medicine. The discovery of mouse embryonic stem cells by Martin Evans 1 for which he was awarded a Nobel Prize in 2007 is notable. The concept of stem cells and regeneration was contemplated as very little and or even absent in adults in several instances. The present understanding seems to have favored the term ‘limited’ or ‘restricted’ ability rather than a blanket absence of ability. It was this belief that probably was one of the inspirations to classify stem cells as embryonic and adult stem cells. The functional distinction given to both these types in an oversimplified language may be put this way. The embryonic stem cell is a less differentiated cell which has a potential for rapid proliferation and differentiation into many cell/ tissue types while the adult stem cells have limited capacity. The obvious advantages of using embryonic stem cells also come with some hiccups. They are feared to produce cancer as well. Cancer Stem cells are believed to be an underlying cause for radio and chemoresitance and even invasive properties of several known cancers. Therefore, the current focus in regenerative medicine is with the use of adult stem cells. Adult stem cells may again be for convenience classed as tissue resident (usually unipotent cells, i.e. principally forms cells of that particular organ in which they reside) and more centralized – multipotent (generalized cells, which may differentiate to a number of cell/tissue types). The stem cells in the brain (neural progenitor cells) located at the dentate gyrus and the sub ventricular zone is an example of a resident stem cell while the stem cells in the bone marrow may be considered as a more generalized cell. To put it simple an organ specific stem cell help regenerate that organ while the other type may help regenerate more than an organ. However this may not a very strict rule for example some studies point to that the stem cells in the liver may act like pancreatic stem cells when the pancreatic function is affected.

Can we create a stem cell? A curious question, for which we already have some clues, cells have been reprogrammed (de-differentiated?) to make them have stem cell like properties. John Gurdon showed that a nucleus of an adult cell can be reprogrammed when placed in an enucleated egg cell (somatic cell nuclear transfer). The resultant cell can initiate normal development. This knowledge was put to practice (surprisingly very soon) by Ian Wilmut and Campbell 2 which resulted in Dolly, the first cloned sheep. Critics may buff this as a very complicated process as if an answer came another astonishing finding in which Kazutoshi Takahashi and Shinya Yamanaka3, 4 in 2006 demonstrated that reprogramming an adult cell is possible by addition of just four transcription factors. These findings however again had the shadow as viral vectors were used for inserting these transcriptional factors. Ever since there has been a flood of activity in this area. Several research groups have demonstrated in a span of just a few months non viral methods of transfer of these factors. The reprogramming of a hepatocyte, B lymphocyte, pancreatic islet beta cells soon followed. The generation of induced pluripotent cells from patients with diseases like amyotrophic lateral sclerosis, adenosine deaminase deficiency-related SCID, Parkinson’s disease and Down’s syndrome are important milestones the field of regenerative medicine has passed.

Stem cells, Regenerative medicine and tissue engineering.

The possibility of a complex organ synthesized or rebuilt has been a major challenge. Perhaps the greatest challenge being the milieu and the three dimensional spatial arrangements of different cell types is the most crucial step towards this direction. Scaffolds for supporting the spatial arrangement of the cells have been evolving.  With rapid strides in the field of biomaterials, especially biodegradable, self assembly polymers, nano materials and the advancements in technologies like electrospinning, molecular self assembly and phase separation used for generation of the modern day biodegradable scaffolds have made the possibilities of synthetic organs stronger today. Three different strategies have been applied for regenerative medicine.

1. Replacing the damaged tissue by injecting cells suspensions or aggregates

2. The transplantation of bioartificial organs synthesized outside the body.

3. In-situ regeneration

 

The proof of concept of their differentiation ability of a stem cell to a particular lineage has been demonstrated by laboratories across the globe. Dopaminergic neurons, oligodentrocyte progenitor cells, motor neurons, pancreatic beta cells, hepatocytes, Retinal cells are some of the examples of specific cell types being produced from embryonic stem cells. However the safety issue especially the risk of teratoma formation and the degrees of functional integration into the tissues are concerns which are not adequately addressed. Induced pluripotent stem cells offer a distinct advantage by virtue of their ability to produce patient specific stem cells. Moreover a variety of adult cells have been reprogrammed to make them multi or pluripotent. Skin fibroblasts, kerationcytes, B lymphocytes, pancreatic beta cells, hepatocytes are a few examples for cell types which has been reprogrammed.

Some of the most important and probable applications may be in the field of chronic diseases, or even to prevent aging or who knows even for eternity!

The practice of stem cells and regenerative medicine

Though the discovery of stem cells date back to Raman V cajol, it use in scientific acceptance came much more recently. The development in the 90’s and the burgeoning of regenerative medicine, a multidisciplinary field is a more recent one.   Hyderabad holds the privilege of of having L.V. Prasad eye institute which perhaps are the world leaders in ‘successful adult stem cell therapy’ for corneal replacement. One of the most accepted procedure is bone marrow transplant. It is widely and effectively used world over. Tissue engineered skin which was approved for clinical use from 1998 by US FDA is in use ever since. Trans tissue migration, vascular and non vascular migration of stem or progenitor cells which are more recently described provides a boost to the regenerative process.                                Bone marrow derived mesenchymal cells have found significant application in human regenerative medicine, for treatment of many maladies like cardiac ischemia, cerebral ischemia, chronic limb ischemia, foot ulcers, chronic knee arthritis. Several diseases like Alzheimer’s, Parkinsonism, muscular dystrophies, refractory systemic lupus erythematosus are possibly other important diseases in which stem cells and regenerative medicine is expected to play a role.    

Stem cells ethical, social and political debates

Does the story of Hela repeat? What if we are able to make a whole human being, is there a possibility of making the human war machines which many of the previous era dictators had dreamt of? The use of embryonic stem cells raised many an eye brows, even the political administrations of some of the most modern countries are having a tough time. Manipulation of embryos, taking of cells has its own social, religious and political implications.

As a general consensus today organ or tissue regeneration are acceptable but not a whole human! However the use of embryonic cells may not be acceptable for many.

As an alternate adult stem cells and induced pluripotent cells are being tipped as the futures for regenerative medicine. Perhaps as per the present understandings, Adult stem cells may be best used for in situ regeneration (repair, regeneration and even improving function of partly damaged organs or tissues) and induced pluripotent cells could serve in organ or tissue engineering where a whole organ or tissue is synthesized outside the host body.

Perhaps the greatest challenge stem cell and regenerative medicine faces is stem cell mobilization, recruitment, finally creation of the microenvironment and functional integration.

To summarize, adult stem cells are used in a number of therapies in humans but are yet not fully defined and its availability is also limited. Embryonic stem cells can be differentiated into almost all adult tissue types but carries the risk of cancer and are not well in control. Significant efforts are being made to produce patient specific stem cells by different techniques. This reprogramming of cells seems to hold some promise for regenerative medicine but we are well behind in understanding them.

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